Targets for therapy in sarcomeric cardiomyopathies

Jil C. Tardiff, Lucie Carrier, Donald M Bers, Corrado Poggesi, Cecilia Ferrantini, Raffaele Coppini, Lars S. Maier, Houman Ashrafian, Sabine Huke, Jolanda Van Der Velden

Research output: Contribution to journalArticle

66 Scopus citations

Abstract

To date, no compounds or interventions exist that treat or prevent sarcomeric cardiomyopathies. Established therapies currently improve the outcome, but novel therapies may be able to more fundamentally affect the disease process and course. Investigations of the pathomechanisms are generating molecular insights that can be useful for the design of novel specific drugs suitable for clinical use. As perturbations in the heart are stage-specific, proper timing of drug treatment is essential to prevent initiation and progression of cardiac disease in mutation carrier individuals. In this review, we emphasize potential novel therapies which may prevent, delay, or even reverse hypertrophic cardiomyopathy caused by sarcomeric gene mutations. These include corrections of genetic defects, altered sarcomere function, perturbations in intracellular ion homeostasis, and impaired myocardial energetics.

Original languageEnglish (US)
Pages (from-to)457-470
Number of pages14
JournalCardiovascular Research
Volume105
Issue number4
DOIs
StatePublished - Apr 1 2015

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Keywords

  • Dilated cardiomyopathy
  • Energetics and microvasculature
  • Gene therapy
  • Hypertrophic cardiomyopathy
  • Ion channels

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Physiology (medical)
  • Physiology

Cite this

Tardiff, J. C., Carrier, L., Bers, D. M., Poggesi, C., Ferrantini, C., Coppini, R., Maier, L. S., Ashrafian, H., Huke, S., & Van Der Velden, J. (2015). Targets for therapy in sarcomeric cardiomyopathies. Cardiovascular Research, 105(4), 457-470. https://doi.org/10.1093/cvr/cvv023